A paper titled “A precision therapy against cancers driven by KIT/PDGFRA mutations” was published online in Science Translational Medicine on November 1, 2017.
“In June 2017, BLU-285 received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation. Previously, the FDA granted orphan drug designation to BLU-285 for the treatment of GIST and SM. The FDA also granted fast track designation to BLU-285 for the treatment of patients with unresectable or metastatic GIST that progressed following treatment with imatinib and a second tyrosine kinase inhibitor and for the treatment of patients with unresectable or metastatic GIST who have the PDGFRα D842V mutation regardless of prior therapy.”
Is a novel, oral, investigational potent pan-KIT and PDGFRα kinase inhibitor with activity across a broad range of tyrosine kinase inhibitor mutations. These include primary KIT exon’s 9,11, or 17 as well as secondary KIT mutations across exon’s 13, 14, 17, and 18. The shape of the DCC-2618 molecule is designed to restore accessibility to the binding pocket when it is blocked by the presence of a loop activation mutation.
A new Phase III Clinical Trial for patients who have been treated with imatinib, sunitinib, and regorafenib was posted to clinicaltrials.gov in November 2017.
Please see the following link for the latest information:
Phase 3 Study of DCC-2618 vs Placebo in Advanced GIST Patients Who Have Been Treated With Prior Anticancer Therapies – Full Text View – ClinicalTrials.gov
This is a 2‑arm, randomized, placebo-controlled, double‑blind, international, multicenter study comparing the efficacy of Deciphera’s DCC-2618 to placebo in patients who have received treatment with prior anticancer therapies.